Full summer beckons for world of health

Good afternoon, health colleagues, and welcome to the European Alliance for Personalised Medicine (EAPM) update, writes EAPM Executive Director Dr. Denis Horgan.

ESMO Congress

The EAPM is busy as ever finalizing articles on a number of topics, in order to lay the framework for engagement in Brussels and at member state level in the coming months. EAPM is also looking forward to the ESMO Congress, the premier oncology congress that will be taking place on 9-12 September, and during which EAPM will be organizing a side event.  

The big pilot to test out the EHDS

The European Health Data Space is arguably the most important piece of legislation for digital health that’s landed in years and its success (or indeed, failure) will affect patients, researchers and policymakers across the bloc. The stakes are high. Yet, despite broad consensus about the need for a big shake-up on health data, it’s not going to be easy to agree on the text. In the face of the challenge, the French Health Data Hub announced this week that it would be leading the pilot that’s set to test out how a system for easier access to health data for research could work. 

It’s one of the first steps toward the EU’s health data space, the bloc’s pitch to let health data flow more freely — to the benefit of both patients who want to access their files when they are abroad, and of researchers and policymakers who want to solve health questions by consulting more data. 

On Monday, the French Health Data Hub announced it had received the go-ahead for an €8 million pilot project focused on one of the two goals of the health data space: the reuse of data for research and policy. The countries involved are France, Belgium, Denmark, Finland, Germany, Hungary, Croatia, Spain and Norway — described by a person involved in the project as “the good students in class.” The Commission didn’t confirm whether it has given the green light.

Laying the foundations

Data exchange is the ultimate goal, but for now, the project needs to address the first step: turning the existing national platforms into the nodes of a bigger, and pan-European, network.

“We are going to build the pipeline, so that it’s possible, technically, to move [the data],” Emmanuel Bacry, chief scientific officer at the French Health Data Hub, said. “We build these pipelines in order to build a network, a European network.”

A critical feature is to have clear guidelines for researchers and policymakers so they know which door to knock on for specific types of data.

The consortium has identified nine possible test cases — ranging from COVID vaccines to cardiometabolic diseases — to run during the two-year pilot. It’s up to the Commission to decide which one will be implemented. After building the infrastructure, some health data might actually move, “if it’s legal and if it’s necessary for the use case,” Bacry stressed.

Those involved in the project know the challenges, with a statement announcing the pilot also acknowledging that they need to address issues with “data quality, access times, lack of interoperability [and] lack of clarity in the legal framework.”

Work in progress

It may seem like a long road to a fully-fledged European platform, where health data flows freely — but in fact, that’s not even the consortium’s target.

“The aim of the project is not to build one single system. So we’re not looking at putting all the data together,” said Petronille Bogaert, project coordinator at Sciensano, the Belgian partner in the consortium. “What we would like to do, however, is to have in each country one single point of entry.”

That goal aligns with the EU’s draft plans, which foresee the establishment of “health data access bodies.” These institutions would be in charge of handing out data permits to researchers and policymakers.

Better data sets

The European Commission intends to adopt implementing regulation to improve the harmonization of health statistics throughout the bloc. That’s what comes out of an answer from Commissioner Kyriakides to a question posed by Cypriot MEP Demetris Papadakis (S&D) regarding the high rate of caesarean births in Cyprus.

Cutting question: MEP Papadakis had asked the Commission whether it intended to take action to promote non-surgical childbirth in the EU, in light of the fact that over 60% of births on the island nation occur through a caesarean, compared with an EU average of 30%. 

HTA decision-making for drugs for rare diseases

Drugs for rare diseases (DRDs) offer important health benefits, but challenge traditional health technology assessment, reimbursement, and pricing processes due to limited effectiveness evidence. Recently, modified processes to address these challenges while improving patient access have been proposed in Canada. 

This review examined processes in 12 jurisdictions to develop recommendations for consideration during formal government-led multi-sectoral discussions currently taking place in Canada. Methods (i) A scoping review of DRD reimbursement processes, (ii) key informant interviews, (iii) a case study of evaluations for and the reimbursement status of a set of 7 DRDs, and (iv) a virtual, multi-stakeholder consultation retreat were conducted. Results Only NHS England has a process specifically for DRDs, while Italy, Scotland, and Australia have modified processes for eligible DRDs. Almost all consider economic evaluations, budget impact analyses, and patient-reported outcomes; but less than half accept surrogate measures. 

Disease severity, lack of alternatives, therapeutic value, quality of evidence, and value for money are factors used in all decision-making processes; only NICE England uses a cost-effectiveness threshold. Budget impact is considered in all jurisdictions except Sweden. In Italy, France, Germany, Spain, and the United Kingdom, specific factors are considered for DRDs. 

However, in all jurisdictions opportunities for clinician/patient input are the same as those for other drugs. Of the 7 DRDs included in the case study, the number that received a positive reimbursement recommendation was highest in Germany and France, followed by Spain and Italy. No relationship between recommendation type and specific elements of the pricing and reimbursement process was found. 

EURORDIS newborn screening

Newborn screening (NBS) is a comprehensive system that includes various elements such as testing the newborn, diagnosis, communication of information to parents, follow-up care and storage of samples for secondary use. NBS is important to people living with a rare disease and their families because for approximately 70% of rare diseases the onset occurs during childhood, but for many diseases clinical signs of symptoms do not appear in the first days or months following birth. 

Recent and continued scientific and technological advancements have opened up the discussion on the expansion of NBS programmes to include rare diseases that could be screened using new sequencing techniques. 

Monkeypox red alert

The World Health Organization has declared the monkeypox outbreak a public health emergency of international concern. The designation is the health body’s highest alert level, underscoring the speed and scale of the outbreak, which counts 16,000 registered cases of the viral disease to date.

The big risk is that the disease — which so far is only endemic in parts of Africa — escapes attempts to control it and becomes established globally. The EU is building a stock of vaccines to try and thwart the spread of infections: “Tried and tested public health measures including strengthened disease surveillance, contact tracing and equitable access to tests, treatments and vaccines for those most at risk is crucial,” said Josie Golding, head of epidemics and epidemiology at the Wellcome Trust. 

“But governments must also support more research to understand why we are seeing new patterns of transmission, evaluate the effectiveness of our current tools and support the development of improved interventions.” Without these, monkeypox could become established in more populations, she warned. 

UK’s NHS ‘in big trouble’ 

Chronic understaffing is a major risk to staff and patient safety, according to a report from the Health and Social Care Committee.

Expert grade: “We could not give the government any higher than an ‘inadequate’ rating on its overall progress in meeting its own targets set for the NHS and social care workforce,” said Jane Dacre, a professor and chair of the expert panel which also published a report today on the government’s commitments in health and social care workforce in England.

The committee’s report spells out just how stretched NHS England is, according to the latest research and figures.

— 12,000: The number of hospital doctors it could be short of.

— Over 50,000: The number of nurses and midwives it could be lacking. 

— Nearly 6.5 million: The record-high number of people awaiting hospital treatment in April. 

A reform of the NHS pension scheme is also needed, the report says: “It is a national scandal that senior doctors are being forced to reduce their working contribution to the NHS or to leave it entirely because of NHS pension arrangements.”

Global HIV efforts faltering through the pandemic, UN report warns

Progress to stop the spread of HIV has continued slipping through the pandemic, a new UN report shows, warning the loss in momentum could continue — and even accelerate — without action.

Though the number of reported infections dropped between 2020 and 2021, the pace of the decline slowed compared to recent years, according to the report. Some regions saw increases in infections for the first time in years. And with people continuing to stay away from the health care system for fear of COVID-19, infections are likely greater than in the official tally.

“The new data revealed in this report are frightening: progress has been faltering, resources have been shrinking and inequalities have been widening,” the report says.

Asia, the most populous region, saw an increase in infections for the first time in a decade. Other regions, including eastern Europe, northern Africa, Latin America and the Middle East, have seen headway in combating the disease slowly over several years.

Political will to fight HIV has wavered along with domestic funding, the report says.

The number of new infections last year — about 1.5 million — was a million over the global goals for the year, representing a major setback in the target of ending AIDS by 2030.

And that is everything from EAPM for now. Stay safe and well, and enjoy the beginning of August.

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